Clinical Trials, Lupus Research, & New Technologies

Clinical trials are crucial to ensure that treatments are safe and effective.

These research studies test the safety of new medical approaches, including the development of new medications and devices (and even combination products such as stents that release medication!).

Clinical trials follow a protocol which is an action plan for the study. The protocol describes:

what the study entails

how it will be conducted

why each portion of the study is necessary

Depending on what is being studied, each clinical trial will have different requirements for participants. Some require volunteers with particular diseases while others require healthy people. For example, age, sex, medication history, and more can be determining factors (or inclusion/exclusion criteria) for a clinical trial.

How are clinical trials monitored and approved?

A clinical trial must be reviewed by an Institutional Review Board (IRB) if it is studying a drug, biological product, or medical device regulated by the Food and Drug Administration (FDA), or if the study is funded or conducted by the federal government.

Institutional Review Boards ensure the safety of studies. For example:

studies are ethical;

participant protections(both their rights and their welfare); and

the risks of the study are reasonable as compared to the potential benefits

IRBs are either private companies (often called independent of commercial IRBs) or affiliated with universities that conduct clinical trials. Physicians, statisticians, and ethicists make up the board members. Above all, there must be at least five members on an IRB with varying backgrounds.

The drug development process

It is expensive and time consuming to bring a new drug or medical device to market. Estimates on cost and timeline vary. However, the Association of Clinical Research Organizations reports that it takes 15 years and as much as $1.2 billion.

Similarly, PhRMA reports that it can take a drug six to seven years simply to complete clinical trials. Also, each success costs an estimated $2.6 billion. Clearly, this is a long, expensive process.

Bringing a medicine to market

The drug development process begins with the discovery phase. The discovery process includes early phase research: scientists identify and investigational drug and perform laboratory tests. After approximately 3 to 6 years of discovery, researchers hope to identify promising drug candidates further study. Then, the formal clinical studies can begin.

Pre-Clinical Research

Duration: 1 – 3 years

Objective: Testing the drug on animals. If the test finds that the product is safe, it is cleared for human clinical trials

Phase 1 – Initial Human Clinical Trials

Duration: 6 months – 1 year

Objective: Test the drug on 20 – 80 healthy individuals. Ensure the drug is safe and generate some pharmacological data

Phase 2 – Expanded Human Clinical Trials

Duration: 1 – 2 years

Objective: Test the drug on 100 – 200 patients who have the disease or condition that the drug was designed for.

Phase 3A

Duration: 2 – 3 years

Objective: Test the drug on several hundred to several thousand participants at numerous sites. Determine the safety and efficacy on a large scale. There is also a focus on regulatory issues.

When complete, the sponsor of the clinical trial submits all relevant data (clinical information, plans for production, packaging, and labeling information) to local regulatory agencies for review. This review can take up to 30 months.

Phase 3B

Duration: 6 months – 1 year

Objective: Test the drug on large groups of patients. Also, evaluate for cost-effectiveness and efficacy as compared to existing approved drugs.

Phase 4

Duration: Ongoing following regulatory approval

Objective: Monitor the drug use after it is approved and ensure that it is safe and effective if used beyond the original application. Additionally, it allows for testing of dosage strengths and formulations and can confirm extra-clinical benefits, like cost-savings or improved quality of life.